Availability of paediatric information in European Medicines Agency approvals.

Lancet Child Adolesc Health

Center for Pediatric Therapeutics and Regulatory Science, and Computational Health Informatics Program, Boston Children's Hospital, Boston, MA, USA; Department of Pediatrics, Harvard Medical School, Boston, MA 02115, USA. Electronic address:

Published: May 2018

Download full-text PDF

Source
http://dx.doi.org/10.1016/S2352-4642(18)30101-9DOI Listing

Publication Analysis

Top Keywords

availability paediatric
4
paediatric european
4
european medicines
4
medicines agency
4
agency approvals
4
availability
1
european
1
medicines
1
agency
1
approvals
1

Similar Publications

There have been disparities reported in prevalence of autism by gender, race, and socioeconomic status with older ages of diagnosis in non-White and in female children. Possible disparities in the ages of autism diagnosis are not well-established within the Military Health System (MHS) pediatric population, where we hypothesized less disparities given universal Tricare coverage for active-duty military families and theoretically equal access to the military treatment facility (MTF). We conducted retrospective cross-sectional analysis using deidentified database repository records from the MHS.

View Article and Find Full Text PDF

Bone marrow transplantation reverses metabolic alterations in multiple sulfatase deficiency: a case series.

Commun Med (Lond)

January 2025

Department of Pediatrics, Division of Blood and Marrow Transplantation, University of Minnesota, Minneapolis, MN, USA.

Background: Multiple sulfatase deficiency (MSD) is an exceptionally rare neurodegenerative disorder due to the absence or deficiency of 17 known cellular sulfatases. The activation of all these cellular sulfatases is dependent on the presence of the formylglycine-generating enzyme, which is encoded by the SUMF1 gene. Disease-causing homozygous or compound heterozygous variants in SUMF1 result in MSD.

View Article and Find Full Text PDF

Evaluating ADHD medication trial representativeness: a Swedish population-based study comparing hypothetically trial-eligible and trial-ineligible individuals.

Lancet Psychiatry

January 2025

Developmental Evidence synthesis, Prediction, Implementation lab, Centre for Innovation in Mental Health, Faculty of Environmental and Life Sciences, University of Southampton, Southampton, UK; Hampshire and Isle of Wight NHS Foundation Trust, Southampton, UK; Clinical and Experimental Sciences (CNS and Psychiatry), Faculty of Medicine, University of Southampton, Southampton, UK; Hassenfeld Children's Hospital at NYU Langone, New York University Child Study Center, New York City, NY, USA; DiMePRe-J-Department of Precision and Regenerative Medicine-Jonic Area, University of Bari Aldo Moro, Bari, Italy.

Background: Randomised controlled trials (RCTs) evaluating ADHD medications often use strict eligibility criteria, potentially limiting generalisability to patients in real-world clinical settings. We aimed to identify the proportion of individuals with ADHD who would be ineligible for medication RCTs and evaluate differences in treatment patterns and clinical and functional outcomes between RCT-eligible and RCT-ineligible individuals.

Methods: We used multiple Swedish national registries to identify individuals with ADHD, aged at least 4 years at the age of diagnosis, initiating pharmacological treatment between Jan 1, 2007, and Dec 31, 2019, with follow-up up to Dec 31, 2020.

View Article and Find Full Text PDF

Maintenance treatment with vedolizumab in paediatric inflammatory bowel disease (VEDOKIDS): 54-week outcomes of a multicentre, prospective, cohort study.

Lancet Gastroenterol Hepatol

January 2025

The Juliet Keidan Institute of Pediatric Gastroenterology and Nutrition, The Eisenberg R&D Authority, Shaare Zedek Medical Center, The Hebrew University of Jerusalem, Jerusalem, Israel. Electronic address:

Background: Infliximab and adalimumab are the only biologics thus far approved for paediatric patients with inflammatory bowel disease (IBD), so other biologics, such as vedolizumab, are prescribed off-label. Despite its frequent use, prospective data for vedolizumab treatment in children are available only for short-term induction outcomes. We aimed to evaluate the long-term efficacy and safety of maintenance therapy with vedolizumab in paediatric patients with IBD.

View Article and Find Full Text PDF

Post-exposure prophylaxis for the prevention of measles: A systematic review.

Vaccine

January 2025

Centre for Immunization Surveillance and Programs, Infectious Disease and Vaccine Program Branch, Public Health Agency of Canada, 130 Colonnade Rd S, Nepean, ON K1A0K9, Canada; Department of Pediatrics, Faculty of Medicine, McGill University. Electronic address:

Background: In susceptible individuals, post-exposure prophylaxis (PEP) is often recommended following exposure to measles, however the data that these recommendations are based on are limited. The objective of this systematic review was to evaluate the efficacy/effectiveness and safety of immunoglobulins (Ig) or measles-containing vaccine (MCV) for the prevention of measles.

Methods: Prospective studies evaluating the use of Ig or MCV as PEP in susceptible individuals exposed to measles were eligible for inclusion.

View Article and Find Full Text PDF

Want AI Summaries of new PubMed Abstracts delivered to your In-box?

Enter search terms and have AI summaries delivered each week - change queries or unsubscribe any time!